The US Food and Drug Administration has approved a second use for the first CRISPR-based medicine, Casgevy, which was approved in December to treat sickle cell disease.

For the β thalassemia trial, 42 patients were evaluated and 39 were still free of the need for red blood cell transfusions and iron chelation after the 12-month period, with the remaining three ...

By Brenda Goodman, CNN (CNN) — The US Food and Drug Administration has approved a second use for the first CRISPR-based medicine, Casgevy, which was approved in December to treat sickle cell ...

The FDA approved a CRISPR-based medicine to treat beta thalassemia, an inherited blood disorder. It’s the same potentially curative therapy cleared by the FDA to treat sickle cell disease.

Beta thalassemia is considered one of the most common rare diseases in the U.S., affecting about 1,500 people nationwide. Because the disorder provides a natural resistance to malaria, it is ...

Exagamglogene autotemcel gave most patients with beta-thalassemia transfusion independence and drastically reduced the number of vaso-occlusive crises in those with sickle cell disease in a pair ...

Achievement supports development of a novel, in vivo approach to treating sickle cell disease and beta thalassemia. CAMBRIDGE, Mass., June 12, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. ...

Beta thalassemia and sickle cell are two red blood cell disorders which both come with massive health implications and shortened lifespans, but at least for UK-based patients the former may soon be… ...

gene edited medicine for the treatment of sickle cell disease and beta thalassemia,” said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine.